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1 天
on MSN
Mom Has 3 Days to Get Son Life-Saving Treatment for Muscular Dystrophy Before He's ...
"My medicine is making me stronger," 7-year-old Hudson Sanford says after receiving a breakthrough gene therapy for Duchenne ...
labiotech
2 天
Six biotechs driving progress in Duchenne muscular dystrophy
Discover six innovative clinical-stage biotech companies fostering R&D in the Duchenne muscular dystrophy therapeutic space.
6 天
新华医院脊柱中心胡苹/傅鑫团队与昆明理工大学陈永昌/季维智团队 ...
动物模型是疾病机制研究和药物开发的重要工具,然而目前已有的小鼠、狗、猪等DMD动物模型均不能很好模拟DMD患者的病程进展。昆明理工大学陈永昌/季维智团队经过多年努力,成功培育出F1代DMD猕猴模型,其中雄性半合子突变个体模拟DMD患者基因型,展现出与 ...
STAT
8 天
Dispute over Duchenne gene therapy highlights thorny access issues
A dispute over a Duchenne gene therapy highlights thorny issues surrounding FDA approvals and insurance policies.
11 天
Wave Life Sciences’ stock soars 51% after biotech posts positive data from Duchenne ...
Biotech reported positive interim data from a mid-stage trial of a treatment for the rare muscle disorder Duchenne muscular ...
10 天
Duchenne Muscular Dystrophy Treatment Drugs, Clinical Trials, Pipeline Insights and ...
Givinostat, is an HDAC inhibitor (HDACi, a principle candidate, currently being developed for the treatment of DMD and BMD.
13 天
再登国际顶级期刊Cell!昆明理工大学陈永昌、季维智团队发表研究成果
日前,昆明理工大学灵长类转化医学研究院、省部共建非人灵长类国家重点实验室、昆明理工大学生命科学与技术学院陈永昌、季维智团队,与广州实验室、上海交通大学医学院附属新华医院等机构合作,应用非人灵长类疾病模型解析杜氏肌营养不良症(Duchenne muscular dystrophy,DMD)发病早期骨骼肌病理变化的关键机制,在国际 ...
charitytoday.co.uk
1 天
The Microscope Ball 2024 raises record amount for Muscular Dystrophy UK
A ‘Relive the 80s’ theme marked the occasion, which brought together 740 members of the commercial property industry at the ...
3 天
on MSN
Manalapan charity left scrambling after NJDOT mix-up nixes Route 9 half-marathon plans
Jar of Hope was all set to stage a half-marathon fundraiser along Route 9 in Western Monmouth County this weekend. Then the ...
9 天
on MSN
Family Life Felt Picture Perfect. Then Our Son, 4, Began Getting Leg Pains
Like many other parents, Theresa and I had never heard of DMD before Michael was diagnosed. It changed everything for us.
1 天
Capricor Therapeutics to Present Long-Term Data from HOPE-2 Open Label Extension Study at ...
Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, announced today that the Company will ...
新华网
12 天
昆工在杜氏肌营养不良症研究中取得新突破
研究结果为DMD发病机制,特别是疾病早期的分子和细胞变化提供了新的见解,揭示了免疫、纤维化以及肌肉干细胞在DMD早期的动态变化,为早期干预和靶向治疗提供了科学依据。此外,DMD猴模型显示FAPs纤维化不依赖于TGFβ通路,为新药研发提供了新的方向。更重要的是,肌肉干细胞功能缺陷导致了肌肉修复障碍,提示DMD是一种干细胞疾病,开发细胞治疗或针对肌肉干细胞进行干预治疗也许是未来研究的重要方向。
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